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Home , Sickle cell disease

BoneA marrow transplantCure! can cure sickle cell disease (SCD).

BloodHere’s is made ofhow three mainit typesworks of cells, each with different jobs: l White cells fight infection l Platelets help clot blood l Red blood cells carry to the body

In sickle cell disease, the red blood cells are sickle-shaped instead of the normal circular shape. This can stop the red blood cells from moving well through your small blood vessels and harm your body throughout your entire life.

All blood cells are made in the . Bone marrow transplant cures a patient with sickle cell disease by replacing their bone marrow, which produces sickle-shaped red blood cells, with bone marrow from a donor that produces healthy circular-shaped red blood cells.

SICKLE CELL DISEASE BONE MARROW TRANSPLANT

Bone Marrow Transplant: A Cure for Sickle Cell Disease For more information, visit www.ChildrensAL.org/sickle-cell-disease Things to Consider About Bone Marrow Transplant

Potential Benefits of Bone Marrow Transplant Risks of Bone Marrow Transplant l Cure Early After Transplant The problems from sickle cell disease such as acute chest l Nausea and vomiting syndrome and new crises will go away after a successful l Hair loss (usually temporary) bone marrow transplant. l Mouth sores l Improved Quality of Life l Need for transfusion of blood and platelets Patients will have more energy and feel better. l Increased risk of infection l Stopping Further Organ Damage l Infertility (there are options to preserve fertility) The organ damage from sickle cell disease such as , l Problems with organs such as heart, liver, lungs or kidneys kidney disease and lung injury will lessen. Later After Transplant l Graft failure This happens when the donor bone marrow does not grow in your child’s body. It is rare. Depending on the strength of the medications used in the preparative regimen, graft failure can result in reoccurrence of sickle cell disease or the need for a second transplant. l Graft versus host disease (GVHD) This occurs when the donor’s white blood cells (infection-fighting cells) attack your child’s body. This can cause rash, diarrhea, liver problems, and muscle problems, dry eyes and many other possible effects. Most cases of GVHD are mild and eventually go away, but some cases are severe and can be life-threatening.

Potential Benefits and Risks The Bone Marrow Transplant Process

Before the Bone Marrow Transplant l The in the preparative regimen will make your child’s l We need to find your child a bone marrow donor. A white blood cell count very low and cause your child to be at risk called HLA typing is done to help match your child with a for infections. possible donor. Possible donors include: Transplant Day Matched Sibling Each full sibling has a 25% chance of being l The donor’s bone marrow is given to your child through a central your child’s HLA match. Survival after bone marrow transplant line. This looks just like a . with a matched sibling is excellent at about 95%. l This can take anywhere from a few minutes to several hours. Matched Unrelated Donor An HLA matched donor that is not l Your child is awake and in his/her own hospital room during the a family member can be found by searching a bone marrow donor bone marrow transplant. registry such as the National Marrow Donor Program (NMDP). l The donor bone marrow moves from the blood vessels directly Haploidentical Donor A family member, such as a parent or into your child’s bone marrow space and begins to grow. sibling, who is not a match, can be a partial HLA match. Research studies are beginning to use these donors for patients After the Transplant with sickle cell disease. l Your child may require blood and platelet transfusions while l Your child will undergo a pre-transplant evaluation to make waiting for the new bone marrow to grow. sure your child is a good candidate for bone marrow transplant. l Your child’s blood counts begin to improve 12-28 days This evaluation includes CT scans, blood work and studies after the transplant. of the kidneys and lungs. l Your child recovers from any side effects from the preparative medications. Hospital Admission and Preparative Regimen l If your child has no major complications, a typical hospital stay l The preparative regimen is a combination of drugs that prepares is 5-6 weeks. your child to receive the donor’s bone marrow. Your child will be admitted to the hospital during the preparative regimen. After Hospital Discharge Medicines are given to weaken your child’s l Your child will still need to take many medications after to create space for the donor’s healthy bone marrow. A special the transplant and be seen in Bone Marrow Transplant (BMT) IV called a central line will be placed to allow these medications clinic 1-3 times weekly. to be given and for blood to be drawn. l Your child will need to stay in the Birmingham area until cleared by l The medicines in the preparative regimen will be given for 7 to 21 the transplant (usually about 3 months after transplant). days prior to your bone marrow transplant. Before, During and After Transplant Is bone marrow transplant anA option for yourCure! child?

Who should consider bone marrow transplant for sickle cell disease? l Patients who have matched sibling donors and have problems from sickle cell disease. l Patients with severe sickle cell disease (prior stroke, admission or multiple pain crises each year). l Patients who have an unrelated donor or a haploidentical donor. l Patients who are eligible for a clinical trial.

Whom should I contact to explore this option for my child? l Call the Pre-Transplant Coordinator, Pediatric Blood and Marrow Transplantation Program, Children's of Alabama, at 205.638.5435.

SICKLE CELL DISEASE BONE MARROW TRANSPLANT

Bone Marrow Transplant: A Cure for Sickle Cell Disease For details, call 205.638.5435